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Efmarodocokin Alfa for Prevention of aGVHD After HSCT

Arslan S, Cutler C, Alousi A, et al. A Phase 1b Study of Efmarodocokin Alfa, an Interleukin-22 Agonist, in Combination With Standard of Care, in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation. Transplantation and Cellular Therapy. 2026; (doi: 10.1016/j.jtct.2026.05.042).

Efmarodocokin alfa, an interleukin-22 agonist, demonstrates potential as prophylaxis against acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic stem cell transplantation (HSCT). With no non-immunosuppressive pharmacologic agent approved for prevention and with other solutions having limited efficacy, aGVHD continues to affect transplant survivors. Findings from a Phase Ib dose-escalation trial, which included 18 participants, highlight the potential of efmarodocokin alfa in this setting when paired with usual prophylactic care. All participants achieved neutrophil engraftment by day 30 post-transplant, regardless of dosing schedule — 30 µg/kg every 4 weeks, 30 µg/kg every 2 weeks, or 60 µg/kg every 2 weeks. No patient developed Grade 3 or 4 aGVHD, although Grade 1 to 2 adverse events were common. The researchers also reported an 83.3% lower gastrointestinal (GI) aGVHD–free survival rate at day 180 and overall survival of 77.8% at 1 year. The evidence supports further investigation into efmarodocokin alfa as a non-immunosuppressive prophylactic strategy against post-HSCT aGVHD, especially in the lower GI tract.

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